New drug aims to stop extra bone formation in rare disease
NCT ID NCT05090891
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This Phase 2 trial tests an experimental drug called INCB000928 (zilurgisertib) in people with fibrodysplasia ossificans progressiva (FOP), a rare genetic condition where soft tissues turn into bone. About 98 participants aged 2 and older will receive either the drug or a placebo daily for several months. The main goal is to see if the drug reduces new abnormal bone growth, which could help control the disease and maintain mobility.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
INCB000928 (zilurgisertib)
What this could lead to
If successful, this drug could slow or prevent the formation of extra bone in people with FOP, potentially reducing disability and improving quality of life.
What could go wrong
This is an early Phase 2 trial with a small number of participants. The drug may not work better than placebo, and side effects are still being studied.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Albert Einstein Israelite Hospital
ACTIVE_NOT_RECRUITINGSão Paulo, 05652-900, Brazil
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Amsterdam Umc - Vu Medisch Centrum (Vumc)
RECRUITINGAmsterdam, 1081 HV, Netherlands
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Ap-Hp Hopital Lariboisiere
RECRUITINGParis, 75010, France
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Beijing Childrens Hospital Capital Medical University
RECRUITINGBeijing, 100045, China
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Centro de Estudios Reumatologicos
ACTIVE_NOT_RECRUITINGSantiago, 7501126, Chile
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Children'S Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Childrens Hospital of Fudan University
RECRUITINGShanghai, 201102, China
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Groote Schuur Hospital Radiation Oncology
ACTIVE_NOT_RECRUITINGCape Town, 07925, South Africa
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Hopital Necker-Enfants Malades
RECRUITINGParis, 75015, France
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Hospital Italiano de Buenos Aires
ACTIVE_NOT_RECRUITINGBuenos Aires, CO 1181, Argentina
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Hospital Universitario Ramon Y Cajal
RECRUITINGMadrid, 28034, Spain
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Instituto Nacional de Rehabilitacion Luis Guillermo Ibarra
ACTIVE_NOT_RECRUITINGTlalpan, 14389, Mexico
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Mayo Clinic Rochester
RECRUITINGRochester, Minnesota, 55905, United States
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Murdoch Children'S Research Institute
ACTIVE_NOT_RECRUITINGParkville, Victoria, 03052, Australia
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Penn Medicine - Perelman Center For Advanced Medicine
ACTIVE_NOT_RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore
ACTIVE_NOT_RECRUITINGRome, 00168, Italy
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Royal National Orthopaedic Hospital
ACTIVE_NOT_RECRUITINGStanmore, HA7 4LP, United Kingdom
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Royal North Shore Hospital
COMPLETEDSt Leonards, New South Wales, 02065, Australia
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Seoul National University Hospital
ACTIVE_NOT_RECRUITINGSeoul, 03080, South Korea
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Shanghai Childrens Medical Center
RECRUITINGShanghai, 200127, China
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Starship Childrens Hospital
ACTIVE_NOT_RECRUITINGAuckland, 01023, New Zealand
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Tongji Hospital of Tongji University
RECRUITINGShanghai, 200065, China
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Uniklinik Koln
RECRUITINGCologne, 50931, Germany
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University Health Network Toronto General Hospital
ACTIVE_NOT_RECRUITINGToronto, Ontario, M5G 2N2, Canada